Cellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD.
Division of Hematology, Children's National Hospital, Washington, DC.
Blood Adv. 2023 Jan 24;7(2):227-234. doi: 10.1182/bloodadvances.2022008137.
Hematopoietic stem cell transplantation (HSCT) is potentially curative for patients with sickle cell disease (SCD). Patients with stable donor engraftment after allogeneic HSCT generally do not experience SCD-related complications; however, there are no published data specifically reporting the change in vaso-occlusive events (VOE) after HSCT. Data regarding the number of VOEs requiring medical attention in the 2 years before allogeneic HSCT were compared with the number of VOEs in the 2 years (0-12 months and 12-24 months) after allogeneic HSCT in patients with SCD. One-hundred sixty-three patients with SCD underwent allogeneic HSCT between 2005 and 2019. The average age at the time of HSCT was 21 years (range, 7 months - 64 years). Most patients underwent nonmyeloablative conditioning (75% [N = 123]) and had a matched sibling donor (72% [N = 118]). The mean number of VOEs was reduced from 5.6 (range, 0-52) in the 2 years before HSCT to 0.9 (range, 0-12) in the 2 years after HSCT (P < .001). Among the post-HSCT events, VOE was more frequent during the first 12 months (0.8 [range, 0-12]) than at 12 to 24 months after HSCT (0.1 [range, 0-8) (P < .001)). In patients who had graft rejection (12%, N = 20), VOEs were reduced from 6.6 (range, 0-24) before HSCT to 1.1 (range, 0-6) and 0.8 (range, 0-8) at 0 to 12 months and 12 to 24 months after HSCT, respectively (P < .001). VOEs requiring medical care were significantly reduced after allogeneic HSCT for patients with SCD. These data will inform the development of novel autologous HSCT gene therapy approaches.
造血干细胞移植 (HSCT) 可能对镰状细胞病 (SCD) 患者具有治愈作用。异基因 HSCT 后稳定供体植入的患者通常不会发生与 SCD 相关的并发症；然而，目前尚无专门报告 HSCT 后血管阻塞性事件 (VOE) 变化的发表数据。将 SCD 患者 HSCT 前 2 年需要医疗关注的 VOE 数量与 HSCT 后 2 年（0-12 个月和 12-24 个月）的 VOE 数量进行比较。2005 年至 2019 年间，163 例 SCD 患者接受了异基因 HSCT。HSCT 时的平均年龄为 21 岁（范围，7 个月至 64 岁）。大多数患者接受非清髓性预处理（75%[N=123]），并接受匹配的同胞供体（72%[N=118]）。HSCT 前 2 年的 VOE 平均数量从 5.6（范围，0-52）减少到 HSCT 后 2 年的 0.9（范围，0-12）（P<.001）。在 HSCT 后的事件中，VOE 在第一个 12 个月（0.8[范围，0-12]）比 HSCT 后 12 至 24 个月（0.1[范围，0-8）更频繁（P<.001）。在发生移植物排斥的患者（12%，N=20）中，VOE 从 HSCT 前的 6.6（范围，0-24）减少到 HSCT 后 0 至 12 个月和 12 至 24 个月的 1.1（范围，0-6）和 0.8（范围，0-8）（P<.001）。SCD 患者接受异基因 HSCT 后，需要医疗护理的 VOE 显著减少。这些数据将为新型自体 HSCT 基因治疗方法的发展提供信息。
